NCT02625246

Safety and Potential Efficacy of Human Mesenchymal Stem Cells in Non-Cystic Fibrosis Bronchiectasis

A Phase I, Trial to Evaluate the Safety, Tolerability, and Potential Efficacy of Allogeneic Human Mesenchymal Stem Cell (hMSC) Infusion in Patients With Non-Cystic Fibrosis Bronchiectasis

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 6 (actual)
• Sponsor: Marilyn Glassberg · Academic / Other
• Sex: All
• Age: 30 Years – 87 Years
• Healthy volunteers: Not accepted

Summary

To demonstrate the safety of bone marrow-derived allogeneic human Mesenchymal Stem Cells (hMSCs) in patients with bronchiectasis receiving standard of care therapy, and to explore treatment efficacy

Detailed description

A Phase 1 investigation will be performed to test the safety of two doses of bone-marrow derived hMSCs (20,000,000 and 100,000,000) administered via peripheral intravenous infusion. Group 1: 3 subjects will receive a single administration of allogeneic hMSCs: 20 x106 (20 million) cells delivered via peripheral intravenous infusion Group 2: 3 subjects will receive a single administration of allogeneic hMSCs: 1 x108 (100 million) cells delivered via peripheral intravenous infusion Interim safety analysis will be performed four weeks after the 1st subject is enrolled in each cohort. Continued safety and tolerability with review of adverse events (AEs) will be assessed at each visit. Efficacy parameters (pulmonary function tests, lung diffusion capacity, lung volumes, 6-Minute Walk Test (6MWT), and dyspnea/Quality of Life (QOL) questionnaires) will be assessed every 12 weeks until study completion. Clinical laboratory tests to assess safety will be performed at every visit. High Resolution Computed Tomography (HRCT) scan will be performed at the baseline visit (if not done within three months prior to enrollment) and then at week 24.

Conditions

• Bronchiectasis

Interventions

Type	Name	Description
BIOLOGICAL	hMSCs	intravenous infusion of bone marrow-derived allogeneic stem cells

Timeline

• Start date: 2016-02-04
• Primary completion: 2019-05-15
• Completion: 2019-05-15
• First posted: 2015-12-09
• Last updated: 2019-08-28

Locations

1 site across 1 country: United States